The American Journal of Managed Care

Guidelines & FDA Approval of Novel Friedreich Ataxia Therapy

A key opinion leader examines the November 2022 guidelines for managing Friedreich's ataxia, discusses the first FDA-approved therapy for the condition, and explains how this treatment is integrated ...
The American Journal of Managed Care

Managing Adverse Events in Novel Friedreich’s Ataxia Therapy

A key opinion leader outlines common adverse events associated with omaveloxolone treatment in Friedreich's ataxia patients and describes strategies used to manage or minimize these adverse effects.
Nasdaq

Solid Biosciences' SGT-212 Gene Therapy for Friedreich's Ataxia Receives FDA IND Clearance, Phase 1b Trial Set for 2025

Solid Biosciences announces FDA clearance for SGT-212, targeting Friedreich’s ataxia with dual gene therapy administration routes, initiating Phase 1b trial in 2025. Solid Biosciences Inc. announced ...
Indianapolis Business Journal

Q&A: Cardiologist has spent career working on treatment for rare condition

Dr. Mark Payne, a professor of pediatrics at the Indiana University School of Medicine and a pediatric cardiologist at Riley ...
Healthline

Treatment Options for Friedreich’s Ataxia

Friedreich’s ataxia is a chronic, progressive neurological condition that leads to impaired muscle coordination. This can affect balance and mobility as well as speech and heart health. Friedreich’s ...
MedCity News

Reata Pharma Drug Wins First FDA Nod in Ultra-Rare Neuromuscular Disorder

Friedrich’s ataxia, an ultra-rare disease characterized by progressively worsening muscle function that ultimately leads to death, now has its first FDA-approved therapy. The regulator has given the ...
Monthly Prescribing Reference

Gene Therapy Candidate Fast Tracked for Friedreich Ataxia Cardiomyopathy

LX2006 (AAVrh.10hFXN) is an adeno-associated virus gene therapy candidate designed to deliver a functional frataxin gene to cardiac cells to improve mitochondrial function. The Food and Drug ...
Nasdaq

Solid Biosciences Receives FDA Fast Track Designation for SGT-212, a Dual Route Gene Therapy for Friedreich’s Ataxia

Solid Biosciences' SGT-212, dual-route gene therapy for Friedreich’s ataxia, receives FDA Fast Track designation for expedited development. Solid Biosciences Inc. has announced that its gene therapy ...
BioWorld

Lexeo, FDA position Friedreich ataxia therapy for accelerated path

Lexeo Therapeutics Inc. feels like it’s in a faster lane to a BLA for its Friedreich ataxia cardiomyopathy gene therapy after talking with the U.S. FDA. The agency told Lexeo that LX-2006 could be on ...
Business Wire

Papillon Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich's Ataxia

SAN DIEGO--(BUSINESS WIRE)--Papillon Therapeutics Inc., a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited ...
Healio

First patient dosed in phase 1/2a trial of Huntington’s, spinocerebellar ataxia therapy

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